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Gene Therapy

Gene Therapy (FLT201) for Gaucher Disease (GALILEO-1 Trial)

Phase 1
Waitlist Available
Research Sponsored by Freeline Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of Gaucher disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis
Treated with either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) and started this treatment at least 2 years prior to dosing with no change in regimen for the prior 3 months. ERT dose ≥15 U/kg and ≤60 U/kg every other week
Must not have
History of splenectomy (partial or total)
Participation in any other clinical study of an investigational medicinal product (IMP), and/or receiving any other IMP during the study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1 (dosing) through the final follow-up visit at week 38
Awards & highlights
No Placebo-Only Group

Summary

This trial will evaluate the safety and effectiveness of a new treatment for Gaucher disease Type 1 in adults.

Who is the study for?
Adults over 18 with Gaucher Disease Type 1, on stable enzyme or substrate therapy for at least 2 years, can join this gene therapy study. They must have low GCase enzyme activity and agree to use contraception. Excluded are those with recent cancer (except certain skin cancers), past gene therapies, bone diseases not due to Gaucher's, severe liver dysfunction, blood disorders unrelated to Gaucher's, certain infections like HIV/Hepatitis C/CMV, allergies to the treatment components or a history of substance abuse.
What is being tested?
FLT201 is being tested for safety and effectiveness in increasing GCase enzyme levels and reducing symptoms in Gaucher Disease Type 1 patients. This first-in-human trial will also explore how different doses affect these outcomes.
What are the potential side effects?
As FLT201 is a new treatment under investigation, potential side effects include immune reactions such as inflammation or allergy-like responses due to the body recognizing it as foreign; however specific side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have Gaucher disease Type 1 with low GCase enzyme activity.
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I've been on the same enzyme or substrate therapy for over 2 years without changes in the last 3 months.
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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had a partial or complete spleen removal.
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I am not currently participating in another clinical trial or taking any investigational medicines.
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I have a history of blood disorders or liver, spleen, bone issues not related to Gaucher disease.
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I am allergic or react badly to the trial medication or its ingredients.
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I have been diagnosed with or suspected to have Type 2 or Type 3 Gaucher disease.
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I have had acute myocarditis or it was found during my screening.
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I have not had cancer, except for certain skin cancers or treated early-stage cancers, in the last 5 years.
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I have tested positive for CMV both through antibody and DNA tests.
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I have a serious liver condition, like cirrhosis.
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I have received gene therapy before.
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I have high blood pressure in the lungs.
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I have not had a live vaccine in the last 12 weeks and do not plan to during the study.
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I have had a bone marrow transplant.
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I have not had serious heart problems in the last 6 months.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 (dosing) through the final follow-up visit at week 38
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 (dosing) through the final follow-up visit at week 38 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of treatment-emergent adverse events

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: FLT201Experimental Treatment1 Intervention
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion.

Find a Location

Who is running the clinical trial?

Freeline TherapeuticsLead Sponsor
6 Previous Clinical Trials
134 Total Patients Enrolled
1 Trials studying Gaucher Disease
75 Patients Enrolled for Gaucher Disease

Media Library

FLT201 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05324943 — Phase 1
Gaucher Disease Research Study Groups: FLT201
Gaucher Disease Clinical Trial 2023: FLT201 Highlights & Side Effects. Trial Name: NCT05324943 — Phase 1
FLT201 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05324943 — Phase 1
~5 spots leftby Dec 2025
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